12-14 December 2023
The U.S. Food and Drug Administration (FDA) issued the final guidance on the Use of Real-World Data (RWD) and Real-World Evidence (RWE) to support regulatory decision-making for drugs and biological products.
The guidance is intended to accelerate medical product development and bring innovations faster and more efficiently to the patients. The agency is issuing this guidance as part of FDA's RWE Program to help support approval of a new indication for a drug already approved or to help support post-approval study requirements. In addition, the guidance clarifies the agency's expectations for sponsors using RWD to support the safety or effectiveness of a drug in clinical studies (including interventional and non-interventional designs). According to the guidance,
The document outlines the agency's general expectations for non-interventional (observational) studies. In these studies, various sources of RWD can be analyzed, including registries, electronic health records (EHRs), and medical claims.
The FDA defines RWD and RWE as follows:
If certain RWD are owned and controlled by other entities, sponsors should have agreements in place with those entities to ensure that relevant patient-level data can be provided to FDA and that source data necessary to verify the RWD are made available for inspection (including all information in original records and certified copies of original records of clinical findings, observations, or other activities in a clinical investigation used for reconstructing and evaluating the investigation). In addition, sponsors should ensure that RWD and associated programming codes and algorithms submitted to FDA are documented, well-annotated, and complete, which would allow FDA to replicate the study analysis using the same dataset and analytic approach. Moreover, the electronic systems used by the sponsor to manage data and produce required records should comply with 21 CFR part 11 (see also FDA's draft guidance Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations: Questions and Answers).
Sponsors who submit non-interventional studies for regulatory review should take responsibility for all activities related to the design, conduct (including data analysis), and oversight of the studies. These activities should include:
The sponsor will retain and make available to the agency upon request a log of any researcher who has significant involvement in the design or conduct of the study (i.e., researcher’s name and affiliations, description of roles or activities performed, qualifications regarding education, training, and experience to perform the proposed study role). If sponsors engage third parties (e.g., data service providers or CROs) to perform certain study-related tasks, sponsors should document the roles and responsibilities of the organization performing the tasks. These documents should be made available to FDA upon request. Sponsors remain responsible for all study-related activities unless a sponsor has transferred its responsibility to a CRO.
For more information please see FDA's final guidance on Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products.